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Reference ID: 4625921 FULL PRESCRIBING INFORMATION ______________ ______________ HIGHLIGHTS OF PRESCRIBING INFORMATION DOSAGE FORMS AND STRENGTHS These highlights do not include all the information needed to use Injection: 12 mg/5 mL (2.4 mg/mL) in a single-dose vial (3) SPINRAZA® safely and effectively. See full prescribing information for ___________________ ____________________ SPINRAZA. CONTRAINDICATIONS None. SPINRAZA (nusinersen) injection, for intrathecal use _______________ _______________ Initial U.S. Approval: 2016 WARNINGS AND PRECAUTIONS __________________ _________________ • Thrombocytopenia and Coagulation Abnormalities: Increased risk for INDICATIONS AND USAGE bleeding complications; testing required at baseline and before each dose SPINRAZA is a survival motor neuron-2 (SMN2)-directed antisense and as clinically needed (5.1, 2.3) oligonucleotide indicated for the treatment of spinal muscular atrophy (SMA) • Renal Toxicity: Quantitative spot urine protein testing required at in pediatric and adult patients (1) baseline and prior to each dose (5.2, 2.3) _______________ ______________ ____________________ ____________________ DOSAGE AND ADMINISTRATION ADVERSE REACTIONS SPINRAZA is administered intrathecally (2.1) The most common adverse reactions that occurred in at least 20% of Dosing Information (2.1) SPINRAZA-treated patients and occurred at least 5% more frequently than in • The recommended dosage is 12 mg (5 mL) per administration control patients were: • Initiate SPINRAZA treatment with 4 loading doses: the first three • lower respiratory infection and constipation in patients with loading doses should be administered at 14-day intervals; the 4th loading infantile-onset SMA (6.1) dose should be administered 30 days after the 3rd dose. A maintenance • pyrexia, headache, vomiting, and back pain in patients with later- dose should be administered once every 4 months thereafter. onset SMA (6.1) Important Preparation and Administration Instructions (2.2) • Allow to warm to room temperature prior to administration To report SUSPECTED ADVERSE REACTIONS, contact Biogen at 1­ • Administer within 4 hours of removal from vial 844-477-4672 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. • Prior to administration, remove 5 mL of cerebrospinal fluid _______________ _______________ USE IN SPECIFIC POPULATIONS • Administer as intrathecal bolus injection over 1 to 3 minutes Pregnancy: Based on animal data, may cause fetal harm (8.1) Laboratory Testing and Monitoring to Assess Safety (2.3) • At baseline and prior to each dose, obtain a platelet count, coagulation See 17 for PATIENT COUNSELING INFORMATION. laboratory testing, and quantitative spot urine protein testing Revised: 06/2020 FULL PRESCRIBING INFORMATION: CONTENTS* 1 INDICATIONS AND USAGE 11 DESCRIPTION 2 DOSAGE AND ADMINISTRATION 12 CLINICAL PHARMACOLOGY 2.1 Dosing Information 12.1 Mechanism of Action 2.2 Important Administration Instructions 12.2 Pharmacodynamics 2.3 Laboratory Testing and Monitoring to Assess Safety 12.3 Pharmacokinetics 3 DOSAGE FORMS AND STRENGTHS 13 NONCLINICAL TOXICOLOGY 4 CONTRAINDICATIONS 13.1 Carcinogenesis, Mutagenesis, Impairment of Fertility 5 WARNINGS AND PRECAUTIONS 14 CLINICAL STUDIES 5.1 Thrombocytopenia and Coagulation Abnormalities 14.1 Infantile-Onset SMA 5.2 Renal Toxicity 14.2 Later-Onset SMA 6 ADVERSE REACTIONS 14.3 Presymptomatic SMA 6.1 Clinical Trials Experience 16 HOW SUPPLIED/STORAGE AND HANDLING 6.2 Immunogenicity 16.1 How Supplied 6.3 Postmarketing Experience 16.2 Storage and Handling 8 USE IN SPECIFIC POPULATIONS 17 PATIENT COUNSELING INFORMATION 8.1 Pregnancy 8.2 Lactation *Sections or subsections omitted from the full prescribing information are not 8.4 Pediatric Use listed. 8.5 Geriatric Use 1 Reference ID: 4625921 FULL PRESCRIBING INFORMATION 1 INDICATIONS AND USAGE SPINRAZA is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. 2 DOSAGE AND ADMINISTRATION 2.1 Dosing Information SPINRAZA is administered intrathecally by, or under the direction of, healthcare professionals experienced in performing lumbar punctures. Recommended Dosage The recommended dosage is 12 mg (5 mL) per administration. Initiate SPINRAZA treatment with 4 loading doses. The first three loading doses should be administered at 14-day intervals. The 4th loading dose should be administered 30 days after the 3rd dose. A maintenance dose should be administered once every 4 months thereafter. Missed Dose If a loading dose is delayed or missed, administer SPINRAZA as soon as possible, with at least 14-days between doses and continue dosing as prescribed. If a maintenance dose is delayed or missed, administer SPINRAZA as soon as possible and continue dosing every 4 months. 2.2 Important Preparation and Administration Instructions SPINRAZA is for intrathecal use only. Prepare and use SPINRAZA according to the following steps using aseptic technique. Each vial is intended for single dose only. Preparation • Store SPINRAZA in the carton in a refrigerator until time of use. o o • Allow the SPINRAZA vial to warm to room temperature (25 C/77 F) prior to administration. Do not use external heat sources. • Inspect the SPINRAZA vial for particulate matter and discoloration prior to administration. Do not administer SPINRAZA if visible particulates are observed or if the liquid in the vial is discolored. The use of external filters is not required. • Withdraw 12 mg (5 mL) of SPINRAZA from the single-dose vial into a syringe and discard unused contents of the vial. • Administer SPINRAZA within 4 hours of removal from vial. Administration • Consider sedation as indicated by the clinical condition of the patient. 2 Reference ID: 4625921 • Consider ultrasound or other imaging techniques to guide intrathecal administration of SPINRAZA, particularly in younger patients. • Prior to administration, remove 5 mL of cerebrospinal fluid. • Administer SPINRAZA as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle [see Dosage and Administration (2.1)]. Do not administer SPINRAZA in areas of the skin where there are signs of infection or inflammation [see Adverse Reactions (6.3)]. 2.3 Laboratory Testing and Monitoring to Assess Safety Conduct the following laboratory tests at baseline and prior to each dose of SPINRAZA and as clinically needed [see Warnings and Precautions (5.1, 5.2)]: • Platelet count • Prothrombin time; activated partial thromboplastin time • Quantitative spot urine protein testing 3 DOSAGE FORMS AND STRENGTHS Injection: 12 mg/5 mL (2.4 mg/mL) nusinersen as a clear and colorless solution in a single-dose vial. 4 CONTRAINDICATIONS None. 5 WARNINGS AND PRECAUTIONS 5.1 Thrombocytopenia and Coagulation Abnormalities Coagulation abnormalities and thrombocytopenia, including acute severe thrombocytopenia, have been observed after administration of some antisense oligonucleotides. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 24 of 146 (16%) SPINRAZA-treated patients with high, normal, or unknown platelet count at baseline developed a platelet level below the lower limit of normal, compared to 10 of 72 (14%) sham- controlled patients. In the sham-controlled study in patients with later-onset SMA (Study 2), two SPINRAZA-treated patients developed platelet counts less than 50,000 cells per microliter, with a lowest level of 10,000 cells per microliter recorded on study day 28. Because of the risk of thrombocytopenia and coagulation abnormalities from SPINRAZA, patients may be at increased risk of bleeding complications. 3 Reference ID: 4625921 Perform a platelet count and coagulation laboratory testing at baseline and prior to each administration of SPINRAZA and as clinically needed. 5.2 Renal Toxicity Renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. SPINRAZA is present in and excreted by the kidney [see Clinical Pharmacology (12.3)]. In the sham-controlled studies for patients with infantile-onset and later-onset SMA, 71 of 123 (58%) of SPINRAZA-treated patients had elevated urine protein, compared to 22 of 65 (34%) sham- controlled patients. Conduct quantitative spot urine protein testing (preferably using a first morning urine specimen) at baseline and prior to each dose of SPINRAZA. For urinary protein concentration greater than 0.2 g/L, consider repeat testing and further evaluation. 6 ADVERSE REACTIONS The following serious adverse reactions are described in detail in other sections of the labeling: • Thrombocytopenia and Coagulation Abnormalities [see Warnings and Precautions (5.1)] • Renal Toxicity [see Warnings and Precautions (5.2)] 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of SPINRAZA cannot be directly compared to rates in clinical trials of other drugs and may not reflect the rates observed in practice. In clinical studies, 346 patients (47% male, 76% Caucasian) were treated with SPINRAZA, including 314 exposed for at least 6 months, 258 exposed for at least 1 year, and 138 exposed for at least 2 years. The safety of SPINRAZA was studied in presymptomatic infants with SMA; pediatric patients (approximately 3 days to 16 years of age at first dose) with symptomatic SMA; in a sham-controlled trial in infants with symptomatic SMA (Study 1; n=80 for SPINRAZA, n=41 for control); in a sham-controlled trial in children with symptomatic SMA (Study 2; n=84 for SPINRAZA, n=42 for control); in
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